1. ¹Departments of Pediatric Hematology/Oncology/Bone Marrow Transplantation and Pathology, University of Colorado School of Medicine, Denver, CO, USA
2. ²Departments of Pediatric Hematology/Oncology/Bone Marrow Transplantation and Pathology, Northwestern University, Chicago, IL, USA
3. ³Departments of Pediatric Hematology/Oncology/Bone Marrow Transplantation and Pathology, University of Florida School of Medicine, Gainesville, FL, USA

Correspondence: Dr RH Giller, The Children's Hospital, Hematology/Oncology/Bone Marrow Transplantation, 1056 East 19th Ave. B115, Denver CO 80218, USA. E-mail: Giller.Roger@tchden.org

Received 12 January 2004; Accepted 6 September 2004; Published online 22 November 2004.

Abstract

Summary:

Pediatric acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) with monosomy 7 is associated with poor disease-free survival when treated by conventional chemotherapy, immunosuppression or supportive measures. Hematopoietic stem cell transplant (HSCT) may improve outcomes; however, data to support this are limited. To better understand the curative potential of HSCT in these patients, all cases of AML and MDS with monosomy 7 treated by two transplant programs (1992 to present) were reviewed. A total of 16 patients were treated, all by allogeneic HSCT. Primary diagnoses were MDS (N=5), therapy-related MDS (N=3), AML (N=5) and therapy-related AML (N=3). In all, 11 patients (69%) survive event-free at 2 years with median follow-up of 986 days (range 330–2011 days). Toxicity caused deaths of the five nonsurviving patients, four of whom were transplanted with active leukemia. Allogeneic HSCT is effective therapy for childhood AML and MDS associated with monosomy 7, particularly for patients with AML in complete remission and MDS.